Gene Transfer for SCID-X1 Using a Self
Inactivating (SIN) Gammaretroviral Vector
A Multi-Institutional Phase I/II Trial Evaluating
the Treatment of SCID-X1 Patients with Retrovirus-mediated Gene Transfer
X-linked severe combined immunodeficiency (SCID-X) is a rare genetic immunodeficiency that prevents a child's
bone marrow from producing infection-fighting white blood cells.
Recent clinical trials
for SCID-X1 documented the efficacy of gene transfer in SCID, although there
was some toxicity related to insertional mutagenesis. This Phase I/II gene
therapy trial for patients with SCID-X1 utilizes a new, safer virus vector
developed collaboratively with investigators around the world.
SCID-X1 Trial Inclusion Criteria
Patients must have a definitive diagnosis of SCID-X1 with no HLA-matched family donors available,
and be either
As of November 2016,
this Phase I/II trial has enrolled seven patients in the United States and six
patients in Europe. Preliminary outcome
data from this trial were published in Hacein-Bey-Abina S, et al. N Engl J Med
2014 Oct 9;371(15):1407-17.